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MX2017014665A - Gene editing of deep intronic mutations. - Google Patents

Gene editing of deep intronic mutations.

Info

Publication number
MX2017014665A
MX2017014665A MX2017014665A MX2017014665A MX2017014665A MX 2017014665 A MX2017014665 A MX 2017014665A MX 2017014665 A MX2017014665 A MX 2017014665A MX 2017014665 A MX2017014665 A MX 2017014665A MX 2017014665 A MX2017014665 A MX 2017014665A
Authority
MX
Mexico
Prior art keywords
crispr
cas
gene editing
deep intronic
intronic mutations
Prior art date
Application number
MX2017014665A
Other languages
Spanish (es)
Inventor
Scaria Abraham
Ruan Guoxiang
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of MX2017014665A publication Critical patent/MX2017014665A/en

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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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Abstract

Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non- naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) - CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self- limiting CRISPER-Cas system.
MX2017014665A 2015-05-16 2016-04-15 Gene editing of deep intronic mutations. MX2017014665A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562162720P 2015-05-16 2015-05-16
PCT/US2016/027987 WO2016186772A2 (en) 2015-05-16 2016-04-15 Gene editing of deep intronic mutations

Publications (1)

Publication Number Publication Date
MX2017014665A true MX2017014665A (en) 2018-04-24

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017014665A MX2017014665A (en) 2015-05-16 2016-04-15 Gene editing of deep intronic mutations.
MX2023006046A MX2023006046A (en) 2015-05-16 2017-11-15 Gene editing of deep intronic mutations.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2023006046A MX2023006046A (en) 2015-05-16 2017-11-15 Gene editing of deep intronic mutations.

Country Status (16)

Country Link
US (2) US11896651B2 (en)
EP (1) EP3298134B8 (en)
JP (3) JP6985250B2 (en)
KR (2) KR20230142649A (en)
CN (3) CN118879692A (en)
AU (2) AU2016265255B2 (en)
BR (1) BR112017024514A2 (en)
CA (1) CA2986021A1 (en)
ES (1) ES2952598T3 (en)
IL (2) IL296365A (en)
MX (2) MX2017014665A (en)
PL (1) PL3298134T3 (en)
PT (1) PT3298134T (en)
RU (2) RU2021132397A (en)
SG (2) SG10201910750WA (en)
WO (1) WO2016186772A2 (en)

Families Citing this family (77)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
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